NCT03389308

Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: Castle Creek Pharmaceuticals, LLC · Industry
• Sex: All
• Age: 6 Months – 99 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101 studies.

Detailed description

This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) who meet all the inclusion/exclusion criteria will be eligible to complete 2 treatment cycles of CCP-020. Each treatment cycle will consist of 8 weeks on treatment (once-daily, at home study medication applications) followed by 8 weeks off treatment (only Investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens) with a maximum of 2 treatment cycles allowed for up to 52 weeks. At Baseline Visit (corresponding to the final study visit of the feeder study), the investigator will perform a clinical assessment and determine if any of the subject's lesions require treatment (up to 30% BSA). If the subject has active lesions as determined by the Investigator's clinical assessment, the subject will initiate a cycle of once-daily, at home study medication application to their EBS lesions for 8 weeks (treatment period) followed by an 8-week period where no treatment will be administered. Subjects presenting with no active lesions (as determined by the Investigator's clinical assessment) will not begin treatment and instead, will be instructed to return to the clinic for re-evaluation in 8 weeks or upon worsening of EBS lesions, whichever happens first.

Conditions

• Epidermolysis Bullosa
• Epidermolysis Bullosa Simplex

Interventions

Type	Name	Description
DRUG	diacerein 1% ointment	Topically apply study medication to lesions identified by the Investigator that require. treatment

Timeline

• Start date: 2017-12-01
• Primary completion: 2020-04-17
• Completion: 2020-04-17
• First posted: 2018-01-03
• Last updated: 2020-09-16
• Results posted: 2020-09-16

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study